Core Viewpoint - Recent advancements in gene editing technology, particularly through pioneering editing techniques, show promise in treating severe neurological diseases, although significant technical and funding challenges remain to be addressed [1][4]. Group 1: Breakthroughs in Gene Editing - Harvard University and Jackson Laboratory successfully utilized pioneering editing technology to correct pathogenic gene mutations in a mouse model of Alternating Hemiplegia of Childhood (AHC), achieving an 85% mutation correction rate [2]. - The treatment led to significant improvements in the mice's brain function, reducing seizure frequency and doubling their lifespan, alongside enhancements in motor and cognitive abilities [2]. - A separate team, led by Professor Qiu Zilong, demonstrated the ability to reverse behavioral abnormalities in MEF2C mutation mice using base editing technology, which is crucial for addressing epilepsy and developmental disorders in children [2][3]. Group 2: Safety and Feasibility - The precision of gene editing technology allows for targeted correction of pathogenic mutations, making it an ideal treatment for neurodevelopmental disorders and autism in children [3]. - The pioneering editing technique requires only a single brain injection for treatment, with minimal off-target effects, confirming its safety and feasibility [3]. - The technology has shown the capability to simultaneously correct five mutations, indicating its broad applicability [3]. Group 3: Challenges Ahead - Despite promising results in mouse models, significant hurdles remain before gene editing can benefit human patients, including the need for advanced delivery systems to target brain cells effectively [4]. - The use of adeno-associated virus 9 (AAV9) as a delivery vehicle poses risks of severe immune reactions at high doses, necessitating the development of improved viral vectors and exploration of non-viral delivery methods [4]. - The biotechnology sector is currently facing a funding crisis, which complicates the lengthy and complex development processes for gene therapies, potentially deterring investors [5].

Core Viewpoint - Researchers at Stanford University have developed a new CRISPR-based technology that enables precise delivery of RNA to specific locations within neurons, potentially revolutionizing the treatment of neurodegenerative diseases and injuries [2][3][4]. Group 1: Technology Development - The new technique, referred to as CRISPR-TO, utilizes CRISPR-Cas13 to target and transport RNA molecules to specific sites within cells, enhancing the ability to repair and regenerate damaged neurons [3][5]. - This method allows for unprecedented precision in RNA delivery, marking a significant advancement in RNA medicine [4][6]. Group 2: Research Findings - Initial tests have shown promising results, with certain RNA molecules increasing the growth of neuronal projections by 50% within 24 hours [5]. - The research team is actively screening additional RNA targets to identify those that can effectively repair neuronal damage in both animals and humans [6]. Group 3: Implications for Treatment - The development of spatial RNA medicine opens new avenues for treating neurodegenerative diseases, emphasizing the importance of RNA localization for therapeutic efficacy [6]. - The technology also holds potential for improving the safety and efficiency of RNA-based drugs by ensuring they are delivered to the correct cellular locations at the right time [6].